Treatment of acromegaly with the growth hormone-receptor antagonist pegvisomant.
AuthorsTrainer, Peter J
Drake, William M
Freda, P U
Van der Lely, A J
Dimaraki, E V
Stewart, P M
Friend, K E
Vance, Mary Lee
Besser, G M
Scarlett, J A
Thorner, Michael O
Powell, J S
Barkan, Ariel L
Sheppard, M C
Rose, D R
Clemmons, David R
Bengtsson, B A
Kleinberg, David L
Cook, D M
Phillips, L S
Strasburger, Christian J
Zib, K A
Bennett, W F
Davis, R J
AffiliationChristie Hospital, Manchester, United Kingdom.
MetadataShow full item record
AbstractBACKGROUND: Patients with acromegaly are currently treated with surgery, radiation therapy, and drugs to reduce hypersecretion of growth hormone, but the treatments may be ineffective and have adverse effects. Pegvisomant is a genetically engineered growth hormone-receptor antagonist that blocks the action of growth hormone. METHODS: We conducted a 12-week, randomized, double-blind study of three daily doses of pegvisomant (10 mg, 15 mg, and 20 mg) and placebo, given subcutaneously, in 112 patients with acromegaly. RESULTS: The mean (+/-SD) serum concentration of insulin-like growth factor I (IGF-I) decreased from base line by 4.0+/-16.8 percent in the placebo group, 26.7+/-27.9 percent in the group that received 10 mg of pegvisomant per day, 50.1+/-26.7 percent in the group that received 15 mg of pegvisomant per day, and 62.5+/-21.3 percent in the group that received 20 mg of pegvisomant per day (P<0.001 for the comparison of each pegvisomant group with placebo), and the concentrations became normal in 10 percent, 54 percent, 81 percent, and 89 percent of patients, respectively (P<0.001 for each comparison with placebo). Among patients treated with 15 mg or 20 mg of pegvisomant per day, there were significant decreases in ring size, soft-tissue swelling, the degree of excessive perspiration, and fatigue. The score fortotal symptoms and signs of acromegaly decreased significantly in all groups receiving pegvisomant (P< or =0.05). The incidence of adverse effects was similar in all groups. CONCLUSIONS: On the basis of these preliminary results, treatment of patients who have acromegaly with a growth hormone-receptor antagonist results in a reduction in serum IGF-I concentrations and in clinical improvement.
CitationTreatment of acromegaly with the growth hormone-receptor antagonist pegvisomant. 2000, 342 (16):1171-7 N. Engl. J. Med.
JournalThe New England Journal of Medicine
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